EDP-297

EDP-297 is a highly potent, selective, steroidal noncarboxylic farnesoid X receptor (FXR) agonist developed for the treatment of nonalcoholic steatohepatitis (NASH). It acts by activating FXR, a nuclear receptor involved in regulating bile acid synthesis, lipid metabolism, and inflammation. FXR activation has been shown to improve metabolic dysfunctions associated with NASH such as hepatic triglyceride accumulation and fibrosis. EDP-297 demonstrated robust anti-fibrotic, anti-inflammatory, and hepatoprotective effects in preclinical models. In phase 1 clinical studies in healthy subjects, EDP-297 showed strong target engagement at low doses with generally good tolerability up to 60 μg daily; pruritus was the most common adverse event at higher doses. The drug was being developed by Enanta Pharmaceuticals but internal development was discontinued after early clinical data suggested no substantial differentiation from earlier candidates; it may still be considered for combination therapies[1][2][3][4][5][6][7][8].